{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,8]],"date-time":"2026-10-08T06:34:41Z","timestamp":1791441281953,"version":"4.3.3"},"reference-count":65,"publisher":"Elsevier BV","issue":"4","license":[{"start":{"date-parts":[[2024,12,1]],"date-time":"2024-12-01T00:00:00Z","timestamp":1733011200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2024,12,1]],"date-time":"2024-12-01T00:00:00Z","timestamp":1733011200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2024,10,31]],"date-time":"2024-10-31T00:00:00Z","timestamp":1730332800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["NS062158"],"award-info":[{"award-number":["NS062158"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100013394","name":"REGENXBIO Inc","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013394","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"published-print":{"date-parts":[[2024,12]]},"DOI":"10.1016\/j.omtm.2024.101369","type":"journal-article","created":{"date-parts":[[2024,11,4]],"date-time":"2024-11-04T18:03:07Z","timestamp":1730743387000},"page":"101369","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":7,"title":["Intrathecal or intravenous AAV9-IDUA\/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS I"],"prefix":"10.1016","volume":"32","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2188-299X","authenticated-orcid":false,"given":"Lalitha R.","family":"Belur","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Avery K.","family":"Huber","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hillary","family":"Mantone","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mason","family":"Robertson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miles C.","family":"Smith","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrea D.","family":"Karlen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kelley F.","family":"Kitto","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Li","family":"Ou","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chester B.","family":"Whitley","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elizabeth","family":"Braunlin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Justin","family":"Furcich","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Troy C.","family":"Lund","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Davis","family":"Seelig","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carolyn A.","family":"Fairbanks","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicholas","family":"Buss","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kwi Hye","family":"Kim","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"R. Scott","family":"McIvor","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2024.101369_bib1","series-title":"The Online Metabolic and Molecular Bases of Inherited Disease","article-title":"The Mucopolysaccharidoses","author":"Neufeld","year":"2019"},{"key":"10.1016\/j.omtm.2024.101369_bib2","first-page":"102","article-title":"Mucopolysaccharidosis type I","volume":"12","author":"Wraith","year":"2014","journal-title":"Pediatr. Endocrinol. Rev."},{"key":"10.1016\/j.omtm.2024.101369_bib3","doi-asserted-by":"crossref","first-page":"161","DOI":"10.3390\/diagnostics10030161","article-title":"Mucopolysaccharidosis Type I","volume":"10","author":"Kubaski","year":"2020","journal-title":"Diagnostics"},{"key":"10.1016\/j.omtm.2024.101369_bib4","doi-asserted-by":"crossref","first-page":"337","DOI":"10.1111\/j.1651-2227.1995.tb13640.x","article-title":"The presenting features of mucopolysaccharidosis type IH (Hurler syndrome)","volume":"84","author":"Cleary","year":"1995","journal-title":"Acta Paediatr."},{"key":"10.1016\/j.omtm.2024.101369_bib5","doi-asserted-by":"crossref","first-page":"112","DOI":"10.1186\/s13023-017-0662-9","article-title":"Open issues in Mucopolysaccharidosis type I-Hurler","volume":"12","author":"Parini","year":"2017","journal-title":"Orphanet J. Rare Dis."},{"key":"10.1016\/j.omtm.2024.101369_bib6","doi-asserted-by":"crossref","first-page":"1838","DOI":"10.3390\/cells9081838","article-title":"Mucopolysaccharidosis Type I: A Review of the Natural History and Molecular Pathology","volume":"9","author":"Hampe","year":"2020","journal-title":"Cells"},{"key":"10.1016\/j.omtm.2024.101369_bib7","article-title":"Enzyme replacement therapy with laronidase (Aldurazyme\u00ae) for treating mucopolysaccharidosis type I","volume":"6","author":"Jameson","year":"2019","journal-title":"Cochrane Database Syst. Rev."},{"key":"10.1016\/j.omtm.2024.101369_bib8","doi-asserted-by":"crossref","first-page":"1566","DOI":"10.2174\/138161208784705504","article-title":"Lysosomal storage diseases and the blood-brain barrier","volume":"14","author":"Begley","year":"2008","journal-title":"Curr. Pharm. Des."},{"key":"10.1016\/j.omtm.2024.101369_bib9","doi-asserted-by":"crossref","first-page":"2697","DOI":"10.2165\/00003495-200767180-00005","article-title":"Treatment of lysosomal storage disorders: progress with enzyme replacement therapy","volume":"67","author":"Rohrbach","year":"2007","journal-title":"Drugs"},{"key":"10.1016\/j.omtm.2024.101369_bib10","doi-asserted-by":"crossref","first-page":"v49","DOI":"10.1093\/rheumatology\/ker396","article-title":"Therapy for the mucopolysaccharidoses","volume":"50","author":"Valayannopoulos","year":"2011","journal-title":"Rheumatology"},{"key":"10.1016\/j.omtm.2024.101369_bib11","doi-asserted-by":"crossref","first-page":"189","DOI":"10.3390\/biom11020189","article-title":"Mucopolysaccharidosis Type I: Current Treatments, Limitations, and Prospects for Improvement","volume":"11","author":"Hampe","year":"2021","journal-title":"Biomolecules"},{"key":"10.1016\/j.omtm.2024.101369_bib12","doi-asserted-by":"crossref","first-page":"S10","DOI":"10.1016\/j.pedneo.2022.10.001","article-title":"Current and new therapies for mucopolysaccharidoses","volume":"64","author":"Penon-Portmann","year":"2023","journal-title":"Pediatr. Neonatol."},{"key":"10.1016\/j.omtm.2024.101369_bib13","doi-asserted-by":"crossref","first-page":"271","DOI":"10.1007\/s10545-016-0006-2","article-title":"Mortality after hematopoietic stem cell transplantation for severe mucopolysaccharidosis type I: the 30-year University of Minnesota experience","volume":"40","author":"Rodgers","year":"2017","journal-title":"J.\u00a0Inherit. Metab. Dis."},{"key":"10.1016\/j.omtm.2024.101369_bib14","doi-asserted-by":"crossref","first-page":"1106","DOI":"10.1016\/j.bbmt.2015.02.011","article-title":"Hematopoietic cell transplantation for mucopolysaccharidosis patients is safe and effective: results after implementation of international guidelines","volume":"21","author":"Aldenhoven","year":"2015","journal-title":"Biol. Blood Marrow Transplant."},{"key":"10.1016\/j.omtm.2024.101369_bib15","doi-asserted-by":"crossref","first-page":"117","DOI":"10.1016\/j.ymgme.2018.11.007","article-title":"Hematopoietic cell transplantation for severe MPS I in the first six months of life: The heart of the matter","volume":"126","author":"Braunlin","year":"2019","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.omtm.2024.101369_bib16","series-title":"Principles and Practice of Lifespan Developmental Neuropsychology","first-page":"427","article-title":"Metabolic and Neurodegenerative Diseases across\u00a0the Life Span","author":"Ziegler","year":"2007"},{"key":"10.1016\/j.omtm.2024.101369_bib17","doi-asserted-by":"crossref","first-page":"1929","DOI":"10.1056\/NEJMoa2106596","article-title":"MPSI Study Group. Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome","volume":"385","author":"Gentner","year":"2021","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2024.101369_bib18","doi-asserted-by":"crossref","first-page":"372","DOI":"10.1016\/S0140-6736(21)02017-1","article-title":"Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1\/2 trial and expanded access","volume":"399","author":"Fumagalli","year":"2022","journal-title":"Lancet"},{"key":"10.1016\/j.omtm.2024.101369_bib19","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1016\/j.nbd.2011.02.015","article-title":"Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I","volume":"43","author":"Wolf","year":"2011","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.omtm.2024.101369_bib20","doi-asserted-by":"crossref","first-page":"618360","DOI":"10.3389\/fnmol.2021.618360","article-title":"Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I","volume":"14","author":"Belur","year":"2021","journal-title":"Front. Mol. Neurosci."},{"key":"10.1016\/j.omtm.2024.101369_bib21","doi-asserted-by":"crossref","first-page":"1088","DOI":"10.1002\/jimd.12414","article-title":"Mucopolysaccharidoses type I gene therapy","volume":"44","author":"Hurt","year":"2021","journal-title":"J.\u00a0Inherit. Metab. Dis."},{"key":"10.1016\/j.omtm.2024.101369_bib22","first-page":"100604","article-title":"Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10","volume":"24","author":"Belur","year":"2020","journal-title":"Mol. Genet. Metab. Rep."},{"key":"10.1016\/j.omtm.2024.101369_bib23","doi-asserted-by":"crossref","first-page":"576","DOI":"10.1089\/hum.2017.187","article-title":"Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I","volume":"28","author":"Belur","year":"2017","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib24","doi-asserted-by":"crossref","first-page":"1298","DOI":"10.1038\/mt.2015.99","article-title":"Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates","volume":"23","author":"Hinderer","year":"2015","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib25","doi-asserted-by":"crossref","first-page":"866","DOI":"10.1016\/j.ymthe.2004.03.011","article-title":"Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene","volume":"9","author":"Hartung","year":"2004","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib26","doi-asserted-by":"crossref","first-page":"14894","DOI":"10.1073\/pnas.1413645111","article-title":"Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I","volume":"111","author":"Hinderer","year":"2014","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2024.101369_bib27","doi-asserted-by":"crossref","first-page":"2018","DOI":"10.1038\/mt.2014.135","article-title":"Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I","volume":"22","author":"Hinderer","year":"2014","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib28","doi-asserted-by":"crossref","first-page":"957","DOI":"10.1089\/hum.2019.012","article-title":"Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys","volume":"30","author":"Hordeaux","year":"2019","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib29","doi-asserted-by":"crossref","first-page":"79","DOI":"10.1016\/j.omtm.2018.06.003","article-title":"Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques","volume":"10","author":"Hordeaux","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2024.101369_bib30","doi-asserted-by":"crossref","first-page":"917","DOI":"10.1038\/sj.gt.3302735","article-title":"Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice","volume":"13","author":"Watson","year":"2006","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib31","doi-asserted-by":"crossref","first-page":"178","DOI":"10.1016\/j.ymthe.2018.10.018","article-title":"ZFN-Mediated In\u00a0Vivo Genome Editing Corrects Murine Hurler Syndrome","volume":"27","author":"Ou","year":"2019","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib32","doi-asserted-by":"crossref","first-page":"8","DOI":"10.1089\/hum.2022.162","article-title":"Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction","volume":"34","author":"Podetz-Pedersen","year":"2023","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib33","doi-asserted-by":"crossref","first-page":"174","DOI":"10.1016\/j.omtm.2019.12.001","article-title":"AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains","volume":"17","author":"Hocquemiller","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2024.101369_bib34","doi-asserted-by":"crossref","first-page":"1211","DOI":"10.1089\/hum.2019.217","article-title":"In\u00a0Vivo Gene Therapy for Mucopolysaccharidosis Type III (Sanfilippo Syndrome): A New Treatment Horizon","volume":"30","author":"Marc\u00f3","year":"2019","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib35","doi-asserted-by":"crossref","first-page":"350","DOI":"10.1089\/hum.2008.155","article-title":"Human \u03b1- iduronidase gene transfer mediated by adeno-associated virus types 1, 2, and 5 in the brain of nonhuman primates: vector diffusion and bio-distribution","volume":"20","author":"Ciron","year":"2009","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib36","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1002\/ana.20150","article-title":"Prevention of neuropathology in the mouse model of Hurler syndrome","volume":"56","author":"Desmaris","year":"2004","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.omtm.2024.101369_bib37","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1038\/mt.2010.265","article-title":"Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes","volume":"19","author":"Ellinwood","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib38","doi-asserted-by":"crossref","first-page":"626","DOI":"10.1089\/hum.2016.184","article-title":"Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System-Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer","volume":"28","author":"Laoharawee","year":"2017","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib39","doi-asserted-by":"crossref","DOI":"10.1016\/j.brainres.2020.146832","article-title":"Comparison of high-dose intracisterna magna and lumbar puncture intrathecal delivery of AAV9 in mice to treat neuropathies","volume":"1739","author":"Bailey","year":"2020","journal-title":"Brain Res."},{"key":"10.1016\/j.omtm.2024.101369_bib40","doi-asserted-by":"crossref","first-page":"411","DOI":"10.1016\/j.ymthe.2019.11.012","article-title":"A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna","volume":"28","author":"Taghian","year":"2020","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib41","doi-asserted-by":"crossref","first-page":"499","DOI":"10.1089\/hum.2021.245","article-title":"Efficacy and Safety of a Krabbe Disease Gene Therapy","volume":"33","author":"Hordeaux","year":"2022","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib42","doi-asserted-by":"crossref","first-page":"1065","DOI":"10.1038\/sj.gt.3302961","article-title":"Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice","volume":"14","author":"Fu","year":"2007","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib43","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1016\/j.omtm.2017.07.004","article-title":"Non-clinical Safety and Efficacy of an AAV2\/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI","volume":"6","author":"Ferla","year":"2017","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2024.101369_bib44","article-title":"Non-invasive intravenous administration of AAV9 transducing iduronate sulfatase leads to global metabolic correction and prevention of neurologic deficits in a mouse model of Hunter syndrome","volume":"34","author":"Laoharawee","year":"2023","journal-title":"Mol. Genet. Metab. Rep."},{"key":"10.1016\/j.omtm.2024.101369_bib45","doi-asserted-by":"crossref","first-page":"5343","DOI":"10.1038\/s41467-021-25697-y","article-title":"Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy","volume":"12","author":"Bertolin","year":"2021","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2024.101369_bib46","doi-asserted-by":"crossref","first-page":"477","DOI":"10.1038\/mt.2014.210","article-title":"Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates","volume":"23","author":"Meyer","year":"2015","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib47","doi-asserted-by":"crossref","first-page":"382","DOI":"10.1089\/hum.2011.200","article-title":"Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates","volume":"23","author":"Samaranch","year":"2012","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib51","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1203\/01.pdr.0000190579.24054.39","article-title":"Cardiac functional and histopathologic findings in humans and mice with mucopolysaccharidosis type I: implications for assessment of therapeutic interventions in hurler syndrome","volume":"59","author":"Braunlin","year":"2006","journal-title":"Pediatr. Res."},{"key":"10.1016\/j.omtm.2024.101369_bib52","first-page":"524","article-title":"Gender-related dimorphism in aortic insufficiency in murine mucopolysaccharidosis type I","volume":"18","author":"Tolar","year":"2009","journal-title":"J.\u00a0Heart Valve Dis."},{"key":"10.1016\/j.omtm.2024.101369_bib53","doi-asserted-by":"crossref","first-page":"193","DOI":"10.1016\/j.ymgme.2022.01.104","article-title":"Contribution of the innate and adaptive immune systems to aortic dilation in murine mucopolysaccharidosis type I","volume":"135","author":"Braunlin","year":"2022","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.omtm.2024.101369_bib54","doi-asserted-by":"crossref","first-page":"480","DOI":"10.1182\/blood-2002-09-2889","article-title":"Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway","volume":"102","author":"Davidoff","year":"2003","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2024.101369_bib55","doi-asserted-by":"crossref","first-page":"4353","DOI":"10.1093\/hmg\/ddv168","article-title":"Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan","volume":"24","author":"Weismann","year":"2015","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtm.2024.101369_bib56","doi-asserted-by":"crossref","first-page":"1470","DOI":"10.1038\/mt.2013.95","article-title":"Mouse gender influences brain transduction by intravascularly administered AAV9","volume":"21","author":"Maguire","year":"2013","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib57","doi-asserted-by":"crossref","DOI":"10.3390\/ijms232012693","article-title":"Sex Difference Leads to Differential Gene Expression Patterns and Therapeutic Efficacy in Mucopolysaccharidosis IVA Murine Model Receiving AAV8 Gene Therapy","volume":"23","author":"Piechnik","year":"2022","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.omtm.2024.101369_bib58","doi-asserted-by":"crossref","first-page":"116","DOI":"10.1016\/j.ymgme.2013.09.008","article-title":"High-dose enzyme replacement therapy in murine Hurler syndrome","volume":"111","author":"Ou","year":"2014","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.omtm.2024.101369_bib59","doi-asserted-by":"crossref","first-page":"1127","DOI":"10.1016\/j.ymthe.2018.03.002","article-title":"Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In\u00a0Vivo Genome Editing","volume":"26","author":"Laoharawee","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib60","article-title":"Gene Therapy Advances: A Meta-Analysis of AAV Usage in Clinical Settings","volume":"8","author":"Au","year":"2021","journal-title":"Front. Med."},{"key":"10.1016\/j.omtm.2024.101369_bib61","doi-asserted-by":"crossref","first-page":"425","DOI":"10.3233\/JND-230122","article-title":"Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry","volume":"11","author":"Servais","year":"2024","journal-title":"J.\u00a0Neuromuscul. Dis."},{"key":"10.1016\/j.omtm.2024.101369_bib62","doi-asserted-by":"crossref","first-page":"1713","DOI":"10.1056\/NEJMoa1706198","article-title":"Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy","volume":"377","author":"Mendell","year":"2017","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2024.101369_bib63","doi-asserted-by":"crossref","first-page":"695","DOI":"10.1089\/hum.2020.182","article-title":"Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy","volume":"31","author":"Wilson","year":"2020","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2024.101369_bib64","doi-asserted-by":"crossref","first-page":"342","DOI":"10.1038\/nm1358","article-title":"Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response","volume":"12","author":"Manno","year":"2006","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2024.101369_bib65","doi-asserted-by":"crossref","first-page":"2215","DOI":"10.1056\/NEJMoa1708538","article-title":"Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant","volume":"377","author":"George","year":"2017","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2024.101369_bib49","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtm.2024.101201","article-title":"Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice","volume":"32","author":"Smith","year":"2024","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2024.101369_bib48","doi-asserted-by":"crossref","first-page":"429","DOI":"10.1016\/j.brainresbull.2007.07.018","article-title":"Characterization of an immunodeficient mouse model of mucopolysaccharidosis type I suitable for preclinical testing of human stem cell and gene therapy","volume":"74","author":"Garcia-Rivera","year":"2007","journal-title":"Brain Res. Bull."},{"key":"10.1016\/j.omtm.2024.101369_bib50","doi-asserted-by":"crossref","first-page":"74","DOI":"10.1037\/h0077579","article-title":"Memory deficits associated with senescence: a neurophysiological and behavioral study in the rat","volume":"93","author":"Barnes","year":"1979","journal-title":"J.\u00a0Comp. Physiol. Psychol."}],"container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050124001852?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050124001852?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,1,23]],"date-time":"2025-01-23T16:06:45Z","timestamp":1737648405000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050124001852"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,12]]},"references-count":65,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2024,12]]}},"alternative-id":["S2329050124001852"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2024.101369","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2024,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Intrathecal or intravenous AAV9-IDUA\/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS I","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2024.101369","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2024 The Authors. Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}],"article-number":"101369"}}